☆ 4.8 Article

National experience with adenosine deaminase deficiency related SCID in Polish children

FRONTIERS IN IMMUNOLOGY (2023)

相关参考文献

注意：仅列出部分参考文献，下载原文获取全部文献信息。

Article Allergy

Hematopoietic cell transplantation in severe combined immunodeficiency: The SCETIDE 2006-2014 European cohort

Arjan C. Lankester et al.

Summary: This comprehensive multicenter analysis examined the outcomes of hematopoietic stem cell transplantation (HSCT) in patients with severe combined immunodeficiency (SCID) and analyzed the factors affecting immune reconstitution (IR) and long-term clinical outcomes. The results showed improved overall survival (OS) and event-free survival (EFS) in all genotypes and donor types. Pretransplantation infections and the use of mismatched related donors were associated with less favorable OS and EFS. The study also identified factors such as specific genetic mutations, conditioning regimen, matched donor HSCT, and levels of naive CD4 T lymphocytes as independent predictors of favorable clinical and immunologic outcomes.

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY (2022)

添加到收藏夹

Article Immunology

\Long-Term Immune Recovery After Hematopoietic Stem Cell Transplantation for ADA Deficiency: a Single-Center Experience

Alexandra Y. Kreins et al.

Summary: Unconditioned HSCT is recommended for ADA-deficient patients with an HLA-matched donor, showing improved survival rates compared to unrelated donors. However, unconditioned procedures may lead to decreased engraftment and metabolic correction, while the use of RIC prior to transplantation can improve long-term outcomes.

JOURNAL OF CLINICAL IMMUNOLOGY (2022)

添加到收藏夹

Article Immunology

Hematopoietic Cell Transplantation for Adenosine Deaminase Severe Combined Immunodeficiency-Improved Outcomes in the Modern Era

Elisabetta Ghimenton et al.

Summary: In the treatment of ADA-SCID patients with HSCT, chemotherapy conditioning, the use of alemtuzumab, and receiving stem cells from unrelated or haploidentical donors contribute to improved survival rates. Results of HSCT have improved since 2007, with no deaths reported and a low incidence of acute graft-versus-host disease (GvHD). Conditioned HSCT with matched unrelated donors has a favorable outcome for ADA-deficient patients.

JOURNAL OF CLINICAL IMMUNOLOGY (2022)

添加到收藏夹

Article Pediatrics

BCG Moreau Polish Substrain Infections in Patients With Inborn Errors of Immunity: 40 Years of Experience in the Department of Immunology, Children's Memorial Health Institute, Warsaw

Ewa Bernatowska et al.

Summary: This study aimed to evaluate the complications of BCG vaccination in patients with Inborn Errors of Immunity (IEI). The results showed that the BCG Moreau vaccine had a superior safety profile compared to other substrains, with significantly fewer cases of disseminated BCG infections (BCGosis) and deaths caused by BCG infection in patients with Severe Combined Immunodeficiency (SCID) and Mendelian susceptibility to mycobacterial diseases (MSMD). The study also confirmed the protecting role of NK cells in preventing BCGosis.

FRONTIERS IN PEDIATRICS (2022)

添加到收藏夹

Article Hematology

Outcomes following treatment for ADA-deficient severe combined immunodeficiency: a report from the PIDTC

Geoffrey D. E. Cuvelier et al.

Summary: Adenosine deaminase (ADA) deficiency is one of the causes of severe combined immune deficiency (SCID). Treatments include enzyme replacement therapy (ERT), hematopoietic cell transplant (HCT), and gene therapy (GT). The study found that patients who underwent ERT before HCT or GT had better outcomes.

BLOOD (2022)

添加到收藏夹

Article Biophysics

EBMT/ESID inborn errors working party guidelines for hematopoietic stem cell transplantation for inborn errors of immunity

A. C. Lankester et al.

BONE MARROW TRANSPLANTATION (2021)

添加到收藏夹

Article Biochemistry & Molecular Biology

Atypical Hemolytic Uremic Syndrome (aHUS) and Adenosine Deaminase (ADA)-Deficient Severe Combined Immunodeficiency (SCID)-Two Diseases That Exacerbate Each Other: Case Report

Anna Bogdal et al.

Summary: Hemolytic uremic syndrome (HUS) and severe combined immunodeficiency (SCID) are two rare but life-threatening diseases that rarely coexist, but may potentially influence the progression of each other. For patients with both conditions, a comprehensive diagnostic and treatment plan is necessary.

INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES (2021)

添加到收藏夹

Article Immunology

Chest Radiographs for Distinguishing ADA-SCID from Other Forms of SCID

Martijn V. Verhagen et al.

JOURNAL OF CLINICAL IMMUNOLOGY (2020)

添加到收藏夹

Article Immunology

Newborn Screening for SCID and Other Severe Primary Immunodeficiency in the Polish-German Transborder Area: Experience From the First 14 Months of Collaboration

Maria Gizewska et al.

FRONTIERS IN IMMUNOLOGY (2020)

添加到收藏夹

Review Allergy

Consensus approach for the management of severe combined immune deficiency caused by adenosine deaminase deficiency

Donald B. Kohn et al.

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY (2019)

添加到收藏夹

Review Immunology

Adenosine Deaminase Deficiency - More Than Just an Immunodeficiency

Kathryn V. Whitmore et al.

FRONTIERS IN IMMUNOLOGY (2016)

添加到收藏夹

Article Allergy

Establishing diagnostic criteria for severe combined immunodeficiency disease (SCID), leaky SCID, and Omenn syndrome: The Primary Immune Deficiency Treatment Consortium experience

William T. Shearer et al.

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY (2014)

添加到收藏夹

Article Hematology

Outcome of hematopoietic stem cell transplantation for adenosine deaminase-deficient severe combined immunodeficiency

Amel Hassan et al.

BLOOD (2012)

添加到收藏夹

Article Allergy

Pulmonary alveolar proteinosis in patients with adenosine deaminase deficiency

Eyal Grunebaum et al.

JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY (2012)

添加到收藏夹

Article Pediatrics

Hyperbilirubinemia and Rapid Fatal Hepatic Failure in Severe Combined Immunodeficiency Caused by Adenosine Deaminase Deficiency (ADA-SCID)

J. S. Kuehl et al.

KLINISCHE PADIATRIE (2011)

添加到收藏夹

Review Hematology

How I treat ADA deficiency

H. Bobby Gaspar et al.

BLOOD (2009)

添加到收藏夹

© Peeref 2019-2024. All rights reserved.